A randomized double blind placebo controlled study of TAK-360 for the treatment of narcolepy with cataplexy

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Takeda Development Center Americas Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Decision date (initial)

2026-06-22

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Takeda Development Center Americas, Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety, Dose response, Pharmacodynamic, Efficacy

To evaluate the safety and tolerability of TAK-360 based on the posttreatment adverse event profile.

Secondary objectives 3

1. 1. To assess the effect of TAK-360 on EDS as measured by sleep latency from the MWT.
2. 2. To assess the effect of TAK-360 on EDS as measured by the ESS total score.
3. 3. To assess the effect of TAK-360 on cataplexy as assessed by WCR.

Conditions and MedDRA coding

Narcolepsy with Cataplexy (NT1)

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

1. 1. The participant is willing and able to understand and fully comply with trial procedures and requirements in the opinion of the investigator.
2. 2. The participant has provided informed consent (that is, in writing, documented via a signed and dated informed consent form (ICF) and any required privacy authorization before the initiation of any trial procedures.
3. 3. The participant is aged 18 to 70 years, inclusive, at the time of signing the ICF. Note: Adult age for the purpose of informed consent may differ by region. Only legal adults should be randomized into the trial
4. 4. The participant has a body mass index within the range 18 to 40 kg/m2 (inclusive).
5. 5. The participant has an International Classification of Sleep Disorders, Third Edition (ICSD-3) or ICSD-3 Text Revision diagnosis of narcolepsy type 1 (narcolepsy with cataplexy; NT1) supported by test results.
6. 6. The participant is positive for the HLA genotype HLA-DQB1*06:02 (positive results for either homozygous or heterozygous alleles will be considered “positive” and acceptable) or results from radioimmunoassay indicate the participant’s CSF OX/hypocretin-1 concentration is ≤110 pg/mL (or less than one-third of the mean values obtained in normal participants within the same standardized assay). Note: Previous HLA results are acceptable if available for review by the investigator and provided for inclusion in the eCRF. In the EU, teh UK, and Switzerland, only previously obtained HLA and/or CSF OX results may be used to confirm eligibility. These historical results should be reviewed by the investigator and provided for inclusion in the eCRF.
7. 7. The participant is judged by the investigator to be sufficiently healthy to participate in the trial, on the basis of clinical evaluations including laboratory safety tests, medical history, physical examination, 12-lead electrocardiogram, and vital sign measurements performed at the screening visit and before the first dose of trial intervention.
8. 8. The participant agrees to follow the contraceptive requirements.
9. 9. In line with country-specific regulations, participants may need to be affiliated with a Social Security Scheme or be a beneficiary of one.
10. 10. The participant has provided informed consent (that is, in writing, documented via a signed and dated ICF) and any required privacy authorization before the initiation of any trial procedures.

Exclusion criteria 23

1. 1. The participant has a current medical disorder, other than narcolepsy with cataplexy, associated with excessive daytime sleepiness.
2. 10. The participant has had major surgery or donated or lost 1 unit of blood (approximately 500 mL) within 4 weeks before the screening visit.
3. 11. The participant is unable to refrain from or anticipates using excluded food products.
4. 12. The participant has participated in another investigational drug trial, in which they received the investigational drug. The interval window from the previous trial will be derived from the date of the last dose of investigational drug in the previous trial to the screening visit of the current trial.
5. 13. The participant plans to participate in any other interventional trial while participating in TAK-360-2004 or has previously participated in another part in TAK-360-2004.
6. 14. The participant has ever discontinued an orexin receptor 2 agonist, including TAK-360, due to a safety or tolerability issue.
7. 15. The participant has a positive test result for hepatitis B surface antigen, hepatitis B core antibody, hepatitis C virus antibody, or HIV antibody/antigen at screening.
8. 16. The participant has a positive pregnancy test result at screening or Day -2 or is breastfeeding.
9. 17. The participant has a positive urine screen result for drugs of abuse and/or positive alcohol test result at screening or Day -2. An exception at screening is made for stimulants or other drugs the participant has been prescribed. Products containing cannabidiol (but not tetrahydrocannabinol) may be allowed throughout the trial, at the discretion of the investigator.
10. 18. The participant is a trial site employee or an immediate family member of or in a dependent relationship with a trial site employee (for example, spouse, parent, child, or sibling) who is involved in the conduct of this trial or may consent under duress.
11. 2. The participant: has a history of myocardial infarction; thyroid disease, coronary artery disease, cardiac rhythm abnormality or heart failure; or an ongoing condition that would preclude enrollment in the view of the investigator.
12. 19. The participant consumes excessive amounts of caffeine
13. 20. The participant currently consumes excessive amounts of alcohol
14. 21. The participant has a usual bedtime later than 1:00 AM, an occupation requiring nighttime shift work or variable shift work within the past 6 months, travel with significant jet lag or plans for travel with significant jet lag
15. 22. The participant, in the opinion of the investigator or subinvestigator, is unlikely to comply with the protocol or is unsuitable for any other reason.
16. 23. The participant is considered to be vulnerable, as defined by local regulations and if exclusion is required by local regulations. Examples of vulnerable persons are persons under safeguard of justice, persons deprived of liberty by judicial or administrative decision, persons receiving psychiatric care without their consent, persons admitted to a health or social establishment for purposes other than research, persons of full age who are subject to a legal protection measure (guardianship or curatorship), and persons unable to express their consent.
17. 3. The participant has current or recent (within 6 months) gastrointestinal disease that is expected to influence the absorption of drugs.
18. 4. The participant has a history of cancer in the past 5 years (does not apply to participants with carcinoma in situ that has been resolved without further treatment or basal cell carcinoma; these participants may be included after approval by the medical monitor).
19. 5. The participant has a clinically significant history of head injury or head trauma.
20. 6. The participant has a history of epilepsy, seizure, or convulsion (except for a single febrile seizure in childhood).
21. 7. The participant has a history of cerebral ischemia, transient ischemic attack (<5 years from screening), intracranial aneurysm, or arteriovenous malformation.
22. 8. The participant has a current history of significant multiple and/or severe allergies (for example, food, drug, or latex allergy) or has had an anaphylactic reaction or significant intolerance to prescription or nonprescription drugs or food which in the investigator’s opinion poses a significant risk to the participant to participate in trial.
23. 9. The participant has a known hypersensitivity to any component of the formulation of TAK-360 or related compounds.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Occurrence of at least 1 TEAE during the trial.

Secondary endpoints 3

1. 1. Parts A and B: Change from baseline in mean sleep latency from the MWT at Week 6.
2. 2. Parts A and B: Change from baseline in ESS total score at Week 6.
3. 3. Parts A and B: WCR at Week 6.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 5

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Takeda Development Center Americas Inc.

Sponsor organisation

Takeda Development Center Americas Inc.

Address

500 Kendall Street

City

Cambridge

Postcode

02142-1108

Country

United States

Scientific contact point

Organisation

Takeda Development Center Americas Inc.

Contact name

Sana Ghafoor

Public contact point

Organisation

Takeda Development Center Americas Inc.

Contact name

Takeda

Third parties 17

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 13 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications